Flex
Pharma, Inc. (NASDAQ:FLKS), a clinical-stage biotechnology company
that is developing innovative and proprietary treatments in Phase 2
randomized, controlled trials for cramps and spasticity associated with
severe neurological diseases such as multiple sclerosis (MS),
Charcot-Marie-Tooth (CMT) and amyotrophic lateral sclerosis (ALS) under
FDA Fast Track designation, today announced positive topline data for
FLX-787 from its randomized, double-blinded, placebo-controlled,
cross-over Australian trial in ALS patients with frequent muscle cramps.
The study was terminated early to focus the Company’s resources on the
ongoing US Phase 2b ALS study (COMMEND).

“We are encouraged by the consistently positive impact of FLX-787 across
multiple efficacy endpoints related to cramping and the associated pain,
despite the small number of patients completing the study,” said Dr.
William McVicar, Flex Pharma President and CEO. “These data demonstrate
the potential for FLX-787 to benefit ALS patients who suffer from
frequent cramping in our ongoing Phase 2b trial, the COMMEND study. With
data readouts in MS, ALS, and CMT expected over the next year, we are
excited to advance the development of FLX-787, under Fast Track
designation for ALS-associated cramping.”

In eight patients who completed the trial per protocol, FLX-787
demonstrated a statistically significant (p<0.05) percentage reduction
from baseline in both cramp-associated pain intensity and stiffness,
relative to placebo control, based on daily patient assessments by
Numerical Rating Scale (NRS). Strong and consistent trends were
demonstrated on multiple endpoints, including: percentage reduction in
the number of cramps from baseline (p=0.08), increase in cramp free days
from baseline (p=0.09), and improvements on both the Patient (PGIC;
p=0.06) and Clinician (CGIC; p=0.06) Global Impression of Change.
FLX-787 was generally well tolerated.

In the patients completing both cross-over periods per protocol:

FLX-787 showed a median 31% reduction in cramps from baseline versus
0.1% reduction for patients while on placebo control;

Patients had a median of 4.4 cramp free days versus 0 for placebo
control;

Patients evaluated themselves as improved with FLX-787 treatment 50%
of the time versus 12.5% with placebo control (PGIC); and

Clinicians blinded to treatments evaluated 50% of patients as improved
with FLX-787 versus 0% for placebo control (CGIC).

The Company also analyzed the Period 1 and Period 2 results of all
patients randomized in the trial and believes the cross-over results are
not driven by a cross-over bias or unblinding effect.

“Nearly all ALS patients report cramps and the majority seek treatment
to relieve their suffering from painful cramping and yet there are no
approved therapies in the US. This data set provides the first clinical
evidence that FLX-787 has an effect in patients with underlying
neurological disease and demonstrates the utility of chemical
neurostimulation in treating symptoms arising from motor neuron
hyperexcitability,” said Flex Pharma Chief Medical Officer Thomas
Wessel, M.D., Ph.D. “In prior studies, FLX-787 has demonstrated similar
efficacy profiles in individuals with normally functioning nervous
systems such as those suffering from nocturnal leg cramps and healthy
normal volunteers studied in our electrically-induced cramp model.”

Data from this study outlined above will be presented at future medical
meetings.

Phase 2 Trial Design

This randomized, blinded, placebo-controlled Phase 2 clinical trial, had
originally planned to enroll up to 60 subjects with ALS or primary
lateral sclerosis (PLS) patients with frequent muscle cramps in
Australia. Due to the challenge of enrolling ALS patients from a limited
population in Australia, and a greater priority placed on the completion
of the larger US Phase 2b trial, the Company announced in July 2017 that
we stopped the trial after 12 patients were randomized. Of these, 8
patients completed both cross-over periods and received both FLX-787 and
the placebo control. Patients were given 19 mg of FLX-787, formulated as
an orally disintegrating tablet (ODT) or placebo control, two or three
times daily. The trial included a 14-day run-in period with no treatment
to establish baseline characteristics, followed by treatment periods
during which patients received FLX-787 or placebo in the first 14-day
treatment period before “crossing-over” to the other treatment for an
additional 14-day treatment period. The exploratory study was designed
to evaluate a number of endpoints relating to cramping frequency,
cramp-associated pain, spasticity, stiffness, global impression of
change by the patient and the clinician, quality of life, sleep and
safety. Eight patients completed both crossover conditions and were
included in the primary per protocol analysis.

About the COMMEND Clinical Trial

The COMMEND trial is an ongoing Phase 2b clinical trial designed to
evaluate FLX-787 in patients with motor neuron disease (MND), focused on
ALS, who suffer from cramps. This randomized, controlled,
double-blinded, parallel design trial in the US includes a 28-day run-in
period to establish a baseline in cramp frequency. Patients are then
randomized to 30 mg of FLX-787 administered three times a day, or
control, for 28 days. Patients will be evaluated for changes in cramp
frequency as the primary endpoint, with a number of secondary endpoints,
including the PGIC, CGIC, cramp-related pain and spasticity.

The Flex Pharma management team will host a conference call and live
webcast with slides with the investment community today, Monday,
November 6, at 8:45 a.m. ET to discuss the information in this press
release.

The live webcast and accompanying slides can be accessed under the
investor relations section of Flex Pharma’s website at www.flex-pharma.com.
A replay of the conference call will be archived under the investor
relations section of the Flex Pharma website for three months after the
call.

About Flex Pharma

Flex Pharma, Inc. is a clinical-stage biotechnology company that is
developing innovative and proprietary treatments in Phase 2 randomized,
controlled trials for cramps and spasticity associated with the severe
neurological diseases of ALS, MS and peripheral neuropathies such as
Charcot-Marie-Tooth (CMT). The Company’s lead candidate, FLX-787, is
being developed under Fast Track designation for the treatment of severe
muscle cramps associated with ALS. Flex Pharma was founded by National
Academy of Science members Rod MacKinnon, M.D. (2003 Nobel Laureate),
and Bruce Bean, Ph.D., recognized leaders in the fields of ion channels
and neurobiology, along with Christoph Westphal, M.D., Ph.D.

Forward-Looking Statements

This press release contains forward-looking statements for purposes of
the safe harbor provisions of the Private Securities Litigation Reform
Act of 1995. Forward-looking statements include statements regarding our
intentions, beliefs, projections, outlook, analyses or current
expectations concerning, among other things, the design and timing of
ongoing and anticipated clinical trials, including the timing for
results of our clinical trials. These forward-looking statements are
based on management's expectations and assumptions as of the date of
this press release and are subject to numerous risks and uncertainties,
which could cause actual results to differ materially from those
expressed or implied by such statements. These risks and uncertainties
include, without limitation: the status, timing, costs, results and
interpretation of our clinical studies; the uncertainties inherent in
conducting clinical studies; our ability to enroll patients in each of
clinical studies on a timely basis; expectations of our ability to make
regulatory filings and obtain and maintain regulatory approvals;
availability of funding sufficient for our foreseeable and unforeseeable
operating expenses and capital expenditure requirements; the inherent
uncertainties associated with intellectual property; and other factors
discussed in greater detail under the heading "Risk Factors" in our
Annual Report on Form 10-K for the year ended December 31, 2016 and
subsequent filings with the Securities and Exchange Commission (SEC).
You are encouraged to read our filings with the SEC, available at www.sec.gov,
for a discussion of these and other risks and uncertainties. Any
forward-looking statements that we make in this press release speak only
as of the date of this press release. We assume no obligation to update
our forward-looking statements whether as a result of new information,
future events or otherwise, after the date of this press release.

BOSTON--(EON: Enhanced Online News)--Click to Tweet Flex Pharma, Inc. (NASDAQ: FLKS), a clinical-stage biotechnology company that is developing innovative and proprietary treatments for cramps and ... more »

BOSTON--(EON: Enhanced Online News)--Click to Tweet Flex Pharma, Inc. (NASDAQ: FLKS), a clinical-stage biotechnology company that is developing innovative and proprietary treatments for cramps and ... more »